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1. (WO2018223073) OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF

Pub. No.:    WO/2018/223073    International Application No.:    PCT/US2018/035712
Publication Date: Fri Dec 07 00:59:59 CET 2018 International Filing Date: Sat Jun 02 01:59:59 CEST 2018
IPC: C07H 21/02
C07H 21/04
C12N 15/113
Applicants: WAVE LIFE SCIENCES LTD.
PFIZER INC.
Inventors: VARGEESE, Chandra
IWAMOTO, Naoki
BUTLER, David Charles Donnell
MARAPPAN, Subramanian
LU, Genliang
ZHANG, Jason Jingxin
VATHIPADIEKAL, Vinod
FRANK-KAMENETSKY, Maria David
APPONI, Luciano Henrique
WISNIEWSKA, Hanna Maria
CHENG, Xiayun
Title: OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
Abstract:
Among other things, the present disclosure provides designed APOC3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.